Hemophilia is a rare blood disorder that primarily affects males, but females can be carriers of the disease. There are two different types of hemophilia carriers: possible carriers and obligate carriers. According to Steps For Living, the mutated genes associated with hemophilia lie on the X chromosome. If a woman…

The U.S. Patent and Trademark Office has granted uniQure a third patent covering the formulation and use of the company’s factor IX Padua gene therapy for the treatment of hemophilia and other bleeding disorders. The new patent (10,465,180) will broaden uniQure’s exclusivity rights over the FIX-Padua encoding gene,…

Three hemophilia B patients said they were able to lead a relatively normal life, including being more active and participating in sports, about three years after receiving the investigational gene therapy AMT-060 in a clinical trial. Their experiences were reported in the study, “The Patient…

Recently, I had the pleasure of speaking with Dr. Carol Kasper, a highly regarded hematologist who has dedicated her life’s work — over 40 years of research and patient care — to individuals with hemophilia. Although Kasper retired three years ago, she continues to…

One year of treatment with Elocta (efmoroctocog alfa) — an extended half-life replacement therapy known as Eloctate in the U.S. — reduced the frequency of bleeds in the elbows and knees of adults with severe hemophilia A, a Spanish study showed. Reduced pain intensity in the elbows also…

Before I became a parent, I never imagined I’d be finding veins and accessing ports. But after my sons were born with severe hemophilia A with inhibitors, my husband and I quickly learned the basics of caregiving, by God’s grace. Over the years, people have said, “Cazandra,…

Fat-shaming is a widespread issue that can also affect people with hemophilia, especially young children. At its core, fat-shaming involves making negative comments or judgments about a person’s weight, which can lead to feelings of inadequacy or embarrassment. While these remarks may be intended to encourage weight loss or…

When given as a prophylactic, or preventive therapy, or on an on-demand basis, FEIBA (factor eight inhibitor bypassing activity) was deemed safe and effective at lessening the number of bleeds in people with severe hemophilia and high levels of inhibitors. That’s according to four-year data from a real-world…

Four patients taking part in a Phase 3 clinical trial of Roche’s investigational hemophilia drug emicizumab (ACE910) have experienced serious thrombotic events, blood clots that cause partial or total obstruction, Reuters reported this week. The patients were being treated for breakthrough bleeding. According to Reuters, a Roche spokesperson said…

uniQure plans to advance its investigational gene therapy AMT-061 into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B. Gene therapies are being develop to treat diseases caused by genetic mutations. It involves replacing the faulty gene in patients with a healthy gene. AMT-061…